A Multi-center, Open Label, Multi-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Tolerance of Gene Therapy Drug GC304 in the Treatment of Primary Hypertriglyceridemia Patients With History of Acute Pancreatitis
The study will evaluate safety and tolerance of intravenous delivery of GC304 gene therapy drug as a treatment of primary hypertriglyceridemic patients with previous onset of acute pancreatitis.
• Diagnosed as primary hypertriglyceridemia poorly managed by regular treatment and dietary control, with episode of acute pancreatitis twice or once of severe acute pancreatitis within 5 years;
• Fasting plasma triglycerides (TG) levels above 5.65 mmol/L (intake of dietary fat \<30 g within 24 hours before blood taken);
• Homozygous or heterozygous mutations in GPIHBP1 or LPL genes by genetic screening;
• The patients within reproductive age take effective contraceptive measures voluntarily entering screening stage until 6 months after the trial;
• The patients fully understand and are able to comply with the requirements of the treatment and are willing to complete the trial as planned, including voluntary compliance with the trial procedures, acceptance of low-fat dietary requirements, and provide of biological samples.
• Be able to understand the procedures and methods of the trial and voluntarily participate with the signature of the informed consent by the patient or his/her guardian.